Nilotinib Compassionate Access Ends

Nilotinib (brand name Tasigna, formerly AMN107) is now FDA-approved and on the market for chronic myelogenous leukemia. FDA guidelines require that Novartis now stop its compassionate-use access program for this drug. The rationale is that patients can get the drug through other avenues, now that it is on the market. The compassionate access program causes extra work for the FDA because it must review each single individual case for approval.

Novartis would prefer that patients first try to enroll in the nilotinib trial for GIST (link here for NCT00471328 ) or else try to get prescription coverage for nilotinib as an off-label indication for GIST. Some insurance companies will cover off-label use for GIST because there are data to suggest that it works. If those two avenues don't work, then patients should contact the Novartis patient assistance program, and they will help get the drug.